Rycals and the RYR-1 Foundation
As you may be aware, Rycals are a new class of drugs that have shown potential benefits in treating patients with RYR-1 muscle disease. Rycals received “Orphan Drug Designation” by the FDA in August 2018.
There is now a Rycal human clinical trial at NIH which is enrolling patients (to learn more about this clinical trial, please click HERE)! This is a major achievement for the RYR-1 Foundation and, more importantly, for the RYR-1 community. We are proud that the RYR-1 Foundation played an important part in this process in a variety of ways: First, in 2016, the RYR-1 Foundation awarded a research grant that evaluated the effect of Rycals on muscle biopsy samples taken from individuals with RYR-1 muscle disease. This study in the laboratory showed that Rycals reversed muscle damage and improved the muscle function in those human samples. The paper summarizing this study can be found by clicking on the image below:
In addition, this study was also the basis for the receipt of a “Bench to Bedside Award” from the NIH. The NIH grant of $150,000 helped defray costs of the human clinical trial. In summary, the initial grant from the RYR-1 Foundation has led to 3 major achievements: 1) Discovery of a new potential treatment for RYR-1 myopathy; 2) Initiation of a human clinical trial; and 3) Receipt of an NIH grant.
Unfortunately, in late 2018, the RYR-1 Foundation was informed that the NIH trial for Rycals was at risk of being permanently terminated due to a personnel problem at NIH. Working closely with the Rycal vendor and our contacts at NIH, we immediately initiated a phone call and letter writing campaign, ultimately soliciting the invaluable assistance of members of the United States Senate and House of Representatives. It is clear that were it not for the successful advocacy efforts of the RYR-1 Foundation, this trial would not be moving forward.